Activity for Genes as Medicine
This activity explores the content and research presented in the short film Genes as Medicine, which tells the story of how scientists succeeded in developing a gene therapy for a type of congenital blindness.
Gene therapy is an approach for treating inherited diseases by delivering corrective versions of genes to patients. Genes as Medicine tells the story of the successes and setbacks of two scientists working to develop a gene therapy for Leber congenital amaurosis, a type of childhood blindness. The film discusses how autosomal recessive conditions are inherited, how scientists use viruses to deliver human genes to cells, and how model organisms are used to test treatments before they are used in patients.
The “Student Handout” probes students’ understanding of the key concepts addressed in the film. The “Educator Materials” document provides suggested pause points in the film with questions for students, background information, and detailed discussion points; a list of related resources and references; and an answer key for the “Student Handout.”
Student Learning Targets
- Explain how gene therapy can be used to treat inherited conditions with known causal mutations.
- Discuss how basic, translational, and clinical research contribute to the development of new medicines.
dog, gene therapy, Leber congenital amaurosis, viral delivery
Acland, Gregory M., Gustavo D. Aguirre, Jharna Ray, Qi Zhang, Tomas S. Aleman, Artur V. Cideciyan, Susan E. Pearce-Kelling, et al. “Gene therapy restores vision in a canine model of childhood blindness.” Nature Genetics 28, 1 (2001): 92–95. https://doi.org/10.1038/ng0501-92.
Russell, Stephen, Jean Bennett, Jennifer A. Wellman, Daniel C. Chung, Zi-Fan Yu, Amy Tillman, Janet Wittes, et al. “Efficacy and safety of voretigene neparvovec (AAV2- hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.” The Lancet 390, 10097 (2017): 849–860. https://doi.org/10.1016/S0140-6736(17)31868-8.
HS-LS1-1, HS-LS3-1, HS-LS3-3; SEP6
IST-1.K, IST-1.P, IST-2.E, IST-3.G, IST-4.A, IST-4.B; SP1
3.4, 3.5, B.4
CC2, CC3; DP1